Craig Mayer, Nick WIlliams, Vojtech Huser
Lister Hill National Center for Biomedical Communications, National Library of Medicine, NIH, Bethesda, MD

See the full regCTG repository at https://github.com/lhncbc/CRI/tree/master/regCTG

To return to the list of conditions and view other reports go to https://lhncbc.github.io/CRI/regCTG/regCTG-condition_report_links.html


Study Counts

Type Study_count
Total_Studies 300
interventional 203
Observational 86
Registry 11

Studies over Time by Start Date

```

Interventional Trials

Intervention Type

intervention_type Study_Count
Drug 88
Drug|placebo 34
Other 19
Device 18
Biological 11
Behavioral 6
Biological|placebo 5
Procedure 5
Genetic 4
Dietary Supplement|placebo 2
Dietary Supplement|placebo|Procedure 2
Drug|Other 2
Behavioral|Dietary Supplement 1
Biological|Procedure 1
Device|Other 1
Device|Other|Procedure 1
Dietary Supplement 1
Genetic|placebo 1
Other|Procedure 1

Count of Studies by Intervention (normalized and mapped)

Interventions over Time

Yearly studies for each intervention by start date

Studies by Country

Country Study_Count
United States 82
Canada 9
France 9
Italy 9
NA 7
United States|Canada 6
Denmark 5
Japan 5
Turkey 4
United Kingdom 4
Germany 3
United States|Australia|Canada|Israel|Sweden|United Kingdom 3
Belgium 2
Belgium|France|Italy|United Kingdom 2
Brazil 2
China 2
Korea, Republic of 2
Switzerland 2
United States|Canada|France|Spain 2
Argentina|Belgium|Brazil|Canada|Chile|Czechia|Denmark|France|Germany|Hungary|Italy|Japan|Korea, Republic of|Netherlands|Norway|Poland|Russian Federation|Spain|Taiwan|Turkey 1
Australia|Belgium|France|Germany|Israel|Netherlands|Spain|Turkey|United Kingdom 1
Belgium|Italy|Netherlands|Sweden 1
Canada|Australia|Belgium|France|Germany|Israel|Italy|Spain|Sweden|United Kingdom 1
France|Italy|Spain|United Kingdom 1
Germany|Netherlands|Spain|Switzerland|United Kingdom 1
Germany|Switzerland 1
Italy|Netherlands 1
Japan|United States|Australia|Brazil|Bulgaria|Canada|China|Hong Kong|India|Jordan|Korea, Republic of|Malaysia|Mexico|Poland|Puerto Rico|Russian Federation|Sri Lanka|Taiwan|Thailand|Turkey 1
Jordan 1
Mexico 1
Netherlands 1
Poland 1
Singapore 1
Spain 1
Sweden 1
United States|Argentina|Australia|Canada|Israel|Italy 1
United States|Australia|Belgium|Brazil|Bulgaria|Canada|Chile|Czechia|France|Germany|Israel|Italy|Korea, Republic of|Poland|Spain|Sweden|Switzerland|Turkey|United Kingdom 1
United States|Australia|Belgium|Brazil|Canada|Chile|Czechia|France|Germany|Israel|Italy|Korea, Republic of|Poland|Spain|Sweden|Switzerland|Turkey|United Kingdom 1
United States|Australia|Belgium|Bulgaria|Canada|Czechia|France|Germany|Greece|Hungary|Israel|Italy|Poland|Spain|Sweden|United Kingdom 1
United States|Australia|Belgium|Canada|Czechia|Greece|Israel|Netherlands|Spain|Sweden|United Kingdom 1
United States|Australia|Belgium|Canada|France|Germany|Israel|Italy|Spain|Sweden|United Kingdom 1
United States|Australia|Canada|Germany|Ireland|Israel|Sweden|United Kingdom 1
United States|Australia|Canada|Germany|Sweden|United Kingdom 1
United States|Austria|Belgium|Bulgaria|France|Germany|Hungary|Ireland|Israel|Italy|Netherlands|Spain|Sweden|Switzerland|United Kingdom 1
United States|Austria|Belgium|France|Germany|Italy|Netherlands|Spain|Sweden|Switzerland 1
United States|Austria|Belgium|France|Germany|Italy|Spain|Switzerland|United Kingdom 1
United States|Belgium|Canada|France|Germany|Israel|Italy|Netherlands|Serbia|Spain|United Kingdom 1
United States|Belgium|Canada|France|Germany|Italy|Netherlands|Spain 1
United States|Belgium|Canada|France|Italy|Netherlands|United Kingdom 1
United States|Belgium|Italy|Spain|United Kingdom 1
United States|Canada|Denmark|France|Germany|Norway|Russian Federation|Sweden 1
United States|Canada|Germany|Italy|United Kingdom 1
United States|Canada|Israel 1
United States|Canada|Italy|Japan|Korea, Republic of|Netherlands|New Zealand|Russian Federation|Spain|Sweden|Taiwan 1
United States|Canada|Japan 1
United States|Denmark|France 1
United States|Denmark|Italy 1
United States|France 1
United States|France|Italy 1
United States|France|Italy|Netherlands 1
United States|France|Italy|United Kingdom 1
United States|Italy|Netherlands 1

Sites per Study

Site_count Study_Count
1 105
2 17
3 7
4 13
5 6
6 7
7 8
8 1
9 4
10 1
11 3
12 3
13 3
14 1
17 3
20 2
21 1
23 2
25 2
27 1
32 1
33 1
37 2
39 1
40 1
44 1
47 1
55 1
58 1
63 1
66 1
86 1

Phase

Phase Study_Count
N/A 56
Phase 2 45
Phase 1 35
Phase 1/Phase 2 29
Phase 3 28
Phase 2/Phase 3 6
Phase 4 3
Early Phase 1 1

Number of Arms

Number_of_Arms Count_of_Studies
1 75
2 75
3 17
4 14
5 3
6 4
9 1
13 1
NA 13

Enrollment Metrics by Phase

Measure Early.Phase.1 N.A Phase.1 Phase.1.Phase.2 Phase.2 Phase.2.Phase.3 Phase.3 Phase.4
Min. 30 4.000 1.00000 3.00000 3.00 19.00000 9.000 11.0
1st Qu. 30 14.000 9.50000 6.00000 15.00 35.25000 64.500 29.5
Median 30 30.000 15.00000 10.00000 21.50 45.00000 131.000 48.0
Mean 30 54.125 16.48571 19.65517 33.00 50.83333 135.963 43.0
3rd Qu. 30 61.000 20.00000 20.00000 45.25 59.25000 216.000 59.0
Max. 30 330.000 66.00000 108.00000 174.00 100.00000 266.000 70.0

Trial Group Type

group_type Group_Count
Experimental 272
Active Comparator 42
Placebo Comparator 42
Other 19
No Intervention 17
NA 13
Sham Comparator 1

Intervention Model

intervention_model Study_Count
Single Group Assignment 94
Parallel Assignment 80
Crossover Assignment 12
Sequential Assignment 9
NA 7
Factorial Assignment 1

Primary Purpose

primary_purpose Study_Count
Treatment 155
Supportive Care 10
Other 8
NA 7
Basic Science 6
Diagnostic 6
Prevention 6
Device Feasibility 2
Health Services Research 2
Screening 1

Observational Studies

Studies by Country

Country Study_Count
United States 44
France 10
Denmark 7
Turkey 6
China 2
NA 2
Norway 2
Austria 1
Denmark|France|United Kingdom 1
Egypt 1
Egypt|India|Lebanon|Lithuania|Romania|Sri Lanka|United Arab Emirates 1
France|United Kingdom 1
Israel 1
Netherlands 1
Switzerland 1
United Kingdom 1
United States|Australia 1
United States|Australia|Canada|Sweden|United Kingdom 1
United States|Australia|Italy 1
United States|Denmark 1

Sites per Study

Site_count Study_Count
1 69
2 7
3 4
5 2
8 1
11 1
12 2

Enrollment Metrics

Measure Observational
Min 4.0000
1st Qu 30.0000
Median 73.0000
Mean 679.4118
3rd Qu 210.0000
Max 20000.0000

Observation Model

observational_model Study_Count
Cohort 42
Case-Only 12
Other 12
Case-Control 6
Family-Based 5
Case Control 4
NA 4
Case-Crossover 1

Time Perspective

time_perspective Study_Count
Prospective 53
Cross-Sectional 15
Other 8
Retrospective 6
NA 4

Registries

Studies by Country

Country Study_Count
United States 4
United Kingdom 3
Egypt 1
France|Austria|Czechia|Germany|Greece|Hungary|Israel|Italy|Latvia|Norway|Portugal|Romania|Slovenia|Sweden|United Kingdom 1
Norway 1
Turkey 1

Sites per Study

Site_count Study_Count
1 8
5 1
51 1
70 1

Enrollment Metrics

Measure Registries
Min 13.000
1st Qu 150.000
Median 327.000
Mean 1370.273
3rd Qu 960.000
Max 10000.000

Registry Model

observational_model Study_Count
Cohort 8
Case-Only 1
Family-Based 1
Other 1

Time Perspective

time_perspective Study_Count
Prospective 10
Cross-Sectional 1

Follow-up

target_duration Study_Count
5 Years 3
6 Years 2
1 Day 1
12 Months 1
20 Years 1
3 Months 1
3 Years 1
40 Years 1

Overview of Studies

Interventional Trials

#If less then 500 trials

nct_id brief_title link overall_status source primary_completion_date
NCT02376816 Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02376816 Completed Nationwide Children’s Hospital 2017-09-30
NCT03406780 A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03406780 Completed Capricor Inc. 2020-03-10
NCT03375255 A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT03375255 Completed Sarepta Therapeutics, Inc. 2019-08-19
NCT03375164 Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03375164 Active, not recruiting Sarepta Therapeutics, Inc. 2021-04-30
NCT03373968 Givinostat in Duchenne’s Muscular Dystrophy Long-term Safety and Tolerability Study https://ClinicalTrials.gov/show/NCT03373968 Recruiting Italfarmaco 2023-12-31
NCT03362502 A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03362502 Recruiting Pfizer 2021-08-26
NCT03340675 Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03340675 Recruiting Cumberland Pharmaceuticals 2023-07-31
NCT03333590 Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03333590 Active, not recruiting Nationwide Children’s Hospital 2020-11-30
NCT03317171 Safety and Efficacy of a Flavonoids- and omega3s-based Compound for the Treatment of Muscular Dystrophies https://ClinicalTrials.gov/show/NCT03317171 Completed Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico 2016-12-31
NCT03238235 Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03238235 Recruiting Italfarmaco 2020-05-31
NCT03236662 (-)- Epicatechin Becker Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03236662 Completed University of California, Davis 2017-11-30
NCT03218995 Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping https://ClinicalTrials.gov/show/NCT03218995 Active, not recruiting Sarepta Therapeutics, Inc. 2021-03-15
NCT03179631 Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03179631 Recruiting PTC Therapeutics 2023-09-05
NCT03167255 Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT03167255 Active, not recruiting NS Pharma, Inc. 2021-05-31
NCT03127241 User-centred Assistive System for Arm Functions in Neuromuscular Subjects https://ClinicalTrials.gov/show/NCT03127241 Completed Politecnico di Milano 2019-11-30
NCT03123913 Study of Testosterone and rHGH in FSHD https://ClinicalTrials.gov/show/NCT03123913 Recruiting University of Rochester 2021-04-30
NCT03067831 Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03067831 Recruiting Stem Cells Arabia 2021-11-30
NCT03038399 Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT03038399 Active, not recruiting ReveraGen BioPharma, Inc. 2020-04-30
NCT03002298 Virtual Reality in Individuals With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03002298 Completed University of Sao Paulo 2016-08-31
NCT02964377 Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents https://ClinicalTrials.gov/show/NCT02964377 Completed University of California, Davis 2018-07-31
NCT02891434 Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02891434 Completed University of Sao Paulo 2016-10-31
NCT02851797 Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02851797 Recruiting Italfarmaco 2022-03-31
NCT02847975 Sodium Nitrate to Improve Blood Flow https://ClinicalTrials.gov/show/NCT02847975 Completed Cedars-Sinai Medical Center 2014-12-31
NCT02836418 Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02836418 Completed aTyr Pharma, Inc. 2017-04-18
NCT02835079 Treatment Effect of Tamoxifen on Patients With DMD https://ClinicalTrials.gov/show/NCT02835079 Active, not recruiting Hadassah Medical Organization 2020-11-30
NCT02834650 Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD https://ClinicalTrials.gov/show/NCT02834650 Recruiting University of California, Los Angeles 2021-01-31
NCT02819557 Study of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02819557 Completed PTC Therapeutics 2018-02-09
NCT02814110 Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02814110 Recruiting Medical University of Bialystok 2020-12-31
NCT02760277 An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT02760277 Completed ReveraGen BioPharma, Inc. 2018-04-26
NCT02760264 A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT02760264 Completed ReveraGen BioPharma, Inc. 2018-05-01
NCT02752048 A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02752048 Completed Taiho Pharmaceutical Co., Ltd. 2017-05-15
NCT02740972 Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT02740972 Completed NS Pharma, Inc. 2018-03-31
NCT02710591 Rimeporide in Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02710591 Completed EspeRare Foundation 2017-12-31
NCT02667483 Study of DS-5141b in Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02667483 Active, not recruiting Daiichi Sankyo, Inc. 2020-12-31
NCT02606136 Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT02606136 Active, not recruiting FibroGen 2020-04-30
NCT02603562 Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02603562 Completed aTyr Pharma, Inc. 2016-12-13
NCT02500381 Study of SRP-4045 and SRP-4053 in DMD Patients https://ClinicalTrials.gov/show/NCT02500381 Recruiting Sarepta Therapeutics, Inc. 2022-05-31
NCT01961557 Evaluating a New Knee-Ankle-Foot Brace to Improve Gait in Children With Movement Disorders https://ClinicalTrials.gov/show/NCT01961557 Recruiting National Institutes of Health Clinical Center (CC) 2023-05-22
NCT02531217 Safety, Tolerability, PK, and Activity of ATYR1940 in Patients With Muscular Dystrophy - Study Extension https://ClinicalTrials.gov/show/NCT02531217 Completed aTyr Pharma, Inc. 2017-03-14
NCT02530905 Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients https://ClinicalTrials.gov/show/NCT02530905 Completed Sarepta Therapeutics, Inc. 2018-10-03
NCT02522507 A Peer E-mentoring Intervention to Improve Employment https://ClinicalTrials.gov/show/NCT02522507 Recruiting Holland Bloorview Kids Rehabilitation Hospital 2019-04-30
NCT02516085 Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin https://ClinicalTrials.gov/show/NCT02516085 Completed University Hospital, Basel, Switzerland 2012-10-31
NCT02485938 HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) https://ClinicalTrials.gov/show/NCT02485938 Completed Capricor Inc. 2017-09-30
NCT02439216 Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02439216 Completed Catabasis Pharmaceuticals 2017-01-12
NCT02434627 Sodium Nitrate for Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02434627 Completed Cedars-Sinai Medical Center 2018-04-30
NCT02432885 Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy Trial https://ClinicalTrials.gov/show/NCT02432885 Completed InCor Heart Institute 2012-06-30
NCT02428673 Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions https://ClinicalTrials.gov/show/NCT02428673 Active, not recruiting Gillette Children’s Specialty Healthcare 2019-03-31
NCT02421523 Strength Training in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02421523 Completed University of Florida 2018-02-15
NCT02420379 Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02420379 Completed Sarepta Therapeutics, Inc. 2018-12-17
NCT02418338 2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children https://ClinicalTrials.gov/show/NCT02418338 Completed University Hospital, Montpellier 2017-06-30
NCT02383511 Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet https://ClinicalTrials.gov/show/NCT02383511 Completed Summit Therapeutics 2015-08-31
NCT02354781 Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02354781 Completed Nationwide Children’s Hospital 2017-11-30
NCT02354352 Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02354352 Completed Ohio State University 2018-05-31
NCT02341053 Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT02341053 Active, not recruiting Gillette Children’s Specialty Healthcare 2019-12-31
NCT02328482 Continuation Protocol to Protocol BBCO-001 https://ClinicalTrials.gov/show/NCT02328482 Completed Bioblast Pharma Ltd. 2017-10-31
NCT02310906 Phase I/II Study of SRP-4053 in DMD Patients https://ClinicalTrials.gov/show/NCT02310906 Completed Sarepta Therapeutics, Inc. 2019-03-25
NCT02295748 An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort https://ClinicalTrials.gov/show/NCT02295748 Completed PTC Therapeutics 2017-08-24
NCT02286947 Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02286947 Completed Sarepta Therapeutics, Inc. 2017-04-21
NCT02251600 A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02251600 Completed PTC Therapeutics 2015-10-31
NCT02246478 A Study of TAS-205 for Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02246478 Completed Taiho Pharmaceutical Co., Ltd. 2015-06-30
NCT02239224 Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients With Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02239224 Completed aTyr Pharma, Inc. 2015-12-21
NCT02235844 Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT02235844 Completed Allergy and Asthma Consultants, Wichita, Kansas 2017-09-30
NCT02196467 Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients https://ClinicalTrials.gov/show/NCT02196467 Recruiting CHU de Quebec-Universite Laval 2022-01-31
NCT02168114 Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle https://ClinicalTrials.gov/show/NCT02168114 Completed University of Florida 2018-01-08
NCT02167217 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02167217 Completed Washington University School of Medicine 2017-02-22
NCT02159963 High Intensity Training in Patients With Facioscapulohumeral Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02159963 Completed Rigshospitalet, Denmark 2015-09-30
NCT02147639 Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02147639 Completed Cedars-Sinai Medical Center 2014-05-31
NCT02090959 An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy https://ClinicalTrials.gov/show/NCT02090959 Completed PTC Therapeutics 2018-06-21
NCT02081625 Exploratory Study of NS-065/NCNP-01 in DMD https://ClinicalTrials.gov/show/NCT02081625 Completed National Center of Neurology and Psychiatry, Japan 2014-11-30
NCT02056808 A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT02056808 Completed Summit Therapeutics 2014-05-31
NCT02018731 L-citrulline and Metformin in Becker’s Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02018731 Completed University Hospital, Basel, Switzerland 2014-09-30
NCT02255552 Study of Eteplirsen in DMD Patients https://ClinicalTrials.gov/show/NCT02255552 Completed Sarepta Therapeutics, Inc. 2019-06-14
NCT02015481 Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients https://ClinicalTrials.gov/show/NCT02015481 Completed Bioblast Pharma Ltd. 2016-04-30
NCT03354039 Tamoxifen in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03354039 Recruiting University Hospital, Basel, Switzerland 2020-06-30
NCT01999075 Stacking Exercises Aid the Decline in FVC and Sick Time https://ClinicalTrials.gov/show/NCT01999075 Completed Children’s Hospital of Eastern Ontario 2018-11-22
NCT01995032 L-citrulline and Metformin in Duchenne’s Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01995032 Completed University Hospital, Basel, Switzerland 2015-10-31
NCT01982695 Cardiomyopathy in DMD: Lisinopril vs. Losartan https://ClinicalTrials.gov/show/NCT01982695 Completed Nationwide Children’s Hospital 2012-08-31
NCT01981915 Optimum Insufflation Capacity in NMD https://ClinicalTrials.gov/show/NCT01981915 Completed Universität Duisburg-Essen 2013-02-28
NCT01976091 Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA https://ClinicalTrials.gov/show/NCT01976091 Completed Nationwide Children’s Hospital 2019-03-14
NCT01954940 Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01954940 Completed Children’s Hospital of Eastern Ontario 2017-03-31
NCT01921374 Mother-caregivers of Children With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01921374 Completed Federal University of São Paulo 2013-08-31
NCT01895283 The Effect of Aerobic Exercise, on Fitness and Functional Muscle Strength, in Patients With Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01895283 Completed Rigshospitalet, Denmark 2014-12-31
NCT01882400 Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01882400 Completed Université de Sherbrooke 2007-09-30
NCT01874275 Duchenne Muscular Dystrophy Clinical Trial https://ClinicalTrials.gov/show/NCT01874275 Completed Alan Neuromedical Technologies, LLC 2014-09-30
NCT01856868 Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study) https://ClinicalTrials.gov/show/NCT01856868 Completed University of California, Davis 2018-09-30
NCT01836627 A Study to Test Lung Stretch Therapy (Hyperinsufflation) in Children With Collagen VI Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01836627 Completed Children’s Hospital Medical Center, Cincinnati 2016-04-30
NCT01826487 Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01826487 Completed PTC Therapeutics 2015-08-31
NCT01826422 Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients https://ClinicalTrials.gov/show/NCT01826422 Completed Coordinación de Investigación en Salud, Mexico 2017-01-31
NCT01823783 Endomysial Fibrosis, Muscular Inflammatory Response and Calcium Homeostasis Dysfunction in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01823783 Recruiting University Hospital, Montpellier 2021-01-31
NCT01805024 Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO) https://ClinicalTrials.gov/show/NCT01805024 Completed Santhera Pharmaceuticals 2017-12-05
NCT01800604 Power Over Pain (POP) Study https://ClinicalTrials.gov/show/NCT01800604 Completed University of Washington 2018-01-31
NCT01790178 Ultrasound in Muscle Biopsy https://ClinicalTrials.gov/show/NCT01790178 Completed Duke University 2015-10-31
NCT01761292 A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD https://ClinicalTrials.gov/show/NCT01761292 Completed Italfarmaco 2014-12-31
NCT01648634 Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01648634 Active, not recruiting Assistance Publique - Hôpitaux de Paris 2021-06-30
NCT01645098 Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01645098 Completed Nationwide Children’s Hospital 2013-04-30
NCT01618331 Protein Supplementation and Exercise in Patients With FSH Muscular Dystrophy- a Randomized Placebo Controlled Study https://ClinicalTrials.gov/show/NCT01618331 Completed Rigshospitalet, Denmark 2013-10-31
NCT01603407 Finding the Optimum Regimen for Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01603407 Completed University of Rochester 2019-11-30
NCT01596803 Effects Antioxidants Supplementation on Muscular Function Patients Facioscapulohumeral Dystrophy (FSHD) https://ClinicalTrials.gov/show/NCT01596803 Completed University Hospital, Montpellier 2012-02-20
NCT01580501 PDE Inhibitors in DMD Study (Acute Dosing Study) https://ClinicalTrials.gov/show/NCT01580501 Completed Cedars-Sinai Medical Center 2013-03-31
NCT01557400 Study of Ataluren for Previously Treated Patients With nmDBMD in Europe, Israel, Australia, and Canada https://ClinicalTrials.gov/show/NCT01557400 Completed PTC Therapeutics 2018-01-31
NCT01540604 CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers https://ClinicalTrials.gov/show/NCT01540604 Completed RSPR Pharma AB NA
NCT01540409 Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01540409 Completed Sarepta Therapeutics, Inc. 2016-04-15
NCT01523964 DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls https://ClinicalTrials.gov/show/NCT01523964 Completed Dart Therapeutics. LLC 2012-08-31
NCT01521546 Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01521546 Completed Ohio State University 2016-06-30
NCT01519349 Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis https://ClinicalTrials.gov/show/NCT01519349 Completed Nationwide Children’s Hospital 2017-10-31
NCT01478022 To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20 https://ClinicalTrials.gov/show/NCT01478022 Completed Parent Project, Italy 2016-03-31
NCT01462292 A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT01462292 Completed GlaxoSmithKline 2013-05-21
NCT01438788 Low Protein Diet in Patients With Collagen VI Related Myopathies https://ClinicalTrials.gov/show/NCT01438788 Completed Istituto Ortopedico Rizzoli 2013-06-30
NCT01396239 Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients https://ClinicalTrials.gov/show/NCT01396239 Completed Sarepta Therapeutics, Inc. 2012-02-29
NCT01393444 ECoG Direct Brain Interface for Individuals With Upper Limb Paralysis https://ClinicalTrials.gov/show/NCT01393444 Completed University of Pittsburgh 2015-04-30
NCT01388764 Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids https://ClinicalTrials.gov/show/NCT01388764 Completed Massachusetts General Hospital 2012-05-31
NCT01359670 Tadalafil and Sildenafil for Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01359670 Completed Cedars-Sinai Medical Center 2013-05-31
NCT01350154 Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients https://ClinicalTrials.gov/show/NCT01350154 Completed Rigshospitalet, Denmark 2013-04-30
NCT01344798 Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C https://ClinicalTrials.gov/show/NCT01344798 Completed Genethon 2010-06-30
NCT01335295 Safety Study of Flavocoxid in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01335295 Completed University of Messina 2013-12-31
NCT04392518 Telerehabilitation in Proximal Muscle Weakness https://ClinicalTrials.gov/show/NCT04392518 Recruiting Koç University 2021-09-30
NCT04313595 Device for Breathing Frequency Monitoring in Muscular Dystrophy https://ClinicalTrials.gov/show/NCT04313595 Completed IRCCS Eugenio Medea 2019-07-15
NCT04313049 LARA: Use of JACO2 Controlled by Joystick or by Voice Control System, in Muscular Dystrophy https://ClinicalTrials.gov/show/NCT04313049 Completed IRCCS Eugenio Medea 2019-11-15
NCT04267354 Arm Cycling in Facioscapulohumeral Dystrophy (FSHD) Patients https://ClinicalTrials.gov/show/NCT04267354 Completed Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust 2018-07-14
NCT04264442 Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE) https://ClinicalTrials.gov/show/NCT04264442 Enrolling by invitation Fulcrum Therapeutics 2024-02-29
NCT04240314 AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications. https://ClinicalTrials.gov/show/NCT04240314 Enrolling by invitation Nationwide Children’s Hospital 2023-01-15
NCT04187482 Myotonic Dystrophy Type 1 Aerobic Exercise Study https://ClinicalTrials.gov/show/NCT04187482 Active, not recruiting McMaster University 2020-07-29
NCT04179409 A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications https://ClinicalTrials.gov/show/NCT04179409 Enrolling by invitation Nationwide Children’s Hospital 2021-09-15
NCT04173234 Aerobic Exercise in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT04173234 Recruiting Hacettepe University 2020-01-15
NCT04154098 Evaluation of a Textile Scapula Orthosis https://ClinicalTrials.gov/show/NCT04154098 Recruiting Swiss Federal Institute of Technology 2020-04-30
NCT04129294 Exploratory Study of NS-089/NCNP-02 in DMD https://ClinicalTrials.gov/show/NCT04129294 Enrolling by invitation National Center of Neurology and Psychiatry, Japan 2021-03-31
NCT04060199 Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) https://ClinicalTrials.gov/show/NCT04060199 Recruiting NS Pharma, Inc. 2024-11-30
NCT04054375 Weekly Steroids in Muscular Dystrophy https://ClinicalTrials.gov/show/NCT04054375 Active, not recruiting Northwestern University 2020-06-01
NCT04004065 Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment https://ClinicalTrials.gov/show/NCT04004065 Recruiting Sarepta Therapeutics, Inc. 2021-08-31
NCT04004000 Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 https://ClinicalTrials.gov/show/NCT04004000 Active, not recruiting Fulcrum Therapeutics 2020-12-31
NCT04003974 Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) https://ClinicalTrials.gov/show/NCT04003974 Active, not recruiting Fulcrum Therapeutics 2020-11-30
NCT03992430 A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON) https://ClinicalTrials.gov/show/NCT03992430 Recruiting Sarepta Therapeutics, Inc. 2024-10-31
NCT03986671 Transmembrane Electromyography (TM-EMG) for the Assessment of Neuromuscular Function in the Oropharynx https://ClinicalTrials.gov/show/NCT03986671 Recruiting Powell Mansfield Inc. 2020-10-31
NCT03985878 A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03985878 Enrolling by invitation Sarepta Therapeutics, Inc. 2026-11-30
NCT03970135 Fat and Glucose Metabolism in Fed and Fasted State in Patients With Low Skeletal Muscle Mass https://ClinicalTrials.gov/show/NCT03970135 Completed Rigshospitalet, Denmark 2017-11-30
NCT03951844 Wearability, Saefty and Usability Assessment for the Upper Limb Exoskeleton BRIDGE/EMPATIA https://ClinicalTrials.gov/show/NCT03951844 Completed Politecnico di Milano 2019-04-02
NCT03936894 Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03936894 Recruiting Children’s National Research Institute 2020-04-30
NCT03917719 An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03917719 Enrolling by invitation Catabasis Pharmaceuticals 2022-06-30
NCT03879304 Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker. https://ClinicalTrials.gov/show/NCT03879304 Enrolling by invitation University of Malaga 2020-12-31
NCT03851107 The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities https://ClinicalTrials.gov/show/NCT03851107 Completed McGill University 2018-05-07
NCT03796637 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren https://ClinicalTrials.gov/show/NCT03796637 Completed PTC Therapeutics 2019-05-24
NCT03789734 Safety Study of BLS-M22 in Healthy Volunteers https://ClinicalTrials.gov/show/NCT03789734 Recruiting BioLeaders Corporation 2020-06-30
NCT03783923 A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) https://ClinicalTrials.gov/show/NCT03783923 Active, not recruiting PTC Therapeutics 2020-11-30
NCT03779646 Bisoprolol in DMD Early Cardiomyopathy https://ClinicalTrials.gov/show/NCT03779646 Recruiting Peking Union Medical College Hospital 2020-07-01
NCT03777319 Spironolactone Versus Prednisolone in DMD https://ClinicalTrials.gov/show/NCT03777319 Recruiting Nationwide Children’s Hospital 2021-12-31
NCT03769116 A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT03769116 Active, not recruiting Sarepta Therapeutics, Inc. 2022-10-10
NCT03760029 A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03760029 Recruiting Pfizer 2022-09-24
NCT03703882 Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03703882 Active, not recruiting Catabasis Pharmaceuticals 2020-06-30
NCT03698149 ECoG BMI for Motor and Speech Control https://ClinicalTrials.gov/show/NCT03698149 Recruiting University of California, San Francisco 2024-08-15
NCT03675126 An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 https://ClinicalTrials.gov/show/NCT03675126 Recruiting Sarepta Therapeutics, Inc. 2024-07-31
NCT03652259 Gene Delivery Clinical Trial of SRP-9003 for Patients With LGMD2E (Beta-sarcoglycan Deficiency) https://ClinicalTrials.gov/show/NCT03652259 Active, not recruiting Sarepta Therapeutics, Inc. 2020-12-31
NCT03648827 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) https://ClinicalTrials.gov/show/NCT03648827 Active, not recruiting PTC Therapeutics 2020-06-30
NCT03627494 First Time in Human (FTIH) Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Repeat Doses of GSK3439171A in Healthy Subjects and to Assess Food Effect https://ClinicalTrials.gov/show/NCT03627494 Completed GlaxoSmithKline 2019-08-26
NCT03611244 Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device https://ClinicalTrials.gov/show/NCT03611244 Recruiting Seoul National University Hospital 2024-12-31
NCT03603288 Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) https://ClinicalTrials.gov/show/NCT03603288 Recruiting Santhera Pharmaceuticals 2023-12-31
NCT02815878 Enhance Wellness for Individuals With Long-Term Physical Disabilities https://ClinicalTrials.gov/show/NCT02815878 Completed University of Washington 2017-11-30
NCT03541070 The Effect of Kinesiology Taping on Balance in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03541070 Completed Hacettepe University 2017-09-15
NCT03531788 Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD https://ClinicalTrials.gov/show/NCT03531788 Active, not recruiting University of Pittsburgh 2020-12-31
NCT03529240 Kinesiology Taping in Duchenne Muscular Dystrophy: Effects on Performance, Gait Characteristics, and Energy Consumption https://ClinicalTrials.gov/show/NCT03529240 Completed Hacettepe University 2017-08-05
NCT03508947 Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03508947 Completed Wave Life Sciences Ltd. 2019-03-06
NCT03490214 Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography https://ClinicalTrials.gov/show/NCT03490214 Completed University of Erlangen-Nürnberg Medical School 2018-08-01
NCT03439670 A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT03439670 Recruiting ReveraGen BioPharma, Inc. 2020-05-01
NCT01247207 Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) https://ClinicalTrials.gov/show/NCT01247207 Enrolling by invitation PTC Therapeutics 2020-12-31
NCT01183767 Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01183767 Completed Charite University, Berlin, Germany 2018-09-06
NCT01153932 Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01153932 Completed GlaxoSmithKline 2012-03-31
NCT01128855 A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects https://ClinicalTrials.gov/show/NCT01128855 Completed GlaxoSmithKline 2011-10-25
NCT01126697 Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies https://ClinicalTrials.gov/show/NCT01126697 Completed Cooperative International Neuromuscular Research Group 2017-12-31
NCT01116570 Physical Training Introduction in Lifestyle of Facioscapulohumeral Dystrophy Patients https://ClinicalTrials.gov/show/NCT01116570 Completed Centre Hospitalier Universitaire de Saint Etienne 2015-01-31
NCT01070511 Tadalafil in Becker Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01070511 Completed Cedars-Sinai Medical Center 2012-12-31
NCT01037309 Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT01037309 Completed BioMarin Pharmaceutical 2013-05-31
NCT01027884 Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT01027884 Completed Santhera Pharmaceuticals 2014-01-31
NCT00912041 BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia https://ClinicalTrials.gov/show/NCT00912041 Recruiting Massachusetts General Hospital 2022-09-30
NCT00873782 Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00873782 Completed University of North Carolina, Chapel Hill 2014-02-28
NCT00866112 A Randomized Exercise Trial for Wheelchair Users https://ClinicalTrials.gov/show/NCT00866112 Completed University of Kansas Medical Center 2011-02-28
NCT03811301 [BrainConnexion] - Neurodevice Phase II Trial https://ClinicalTrials.gov/show/NCT03811301 Recruiting National Neuroscience Institute 2021-12-31
NCT00821548 Electrostimulation of Shoulder Girdle and Quadriceps Muscles in Facioscapulohumeral Muscular Dystrophy Patients https://ClinicalTrials.gov/show/NCT00821548 Completed Centre Hospitalier Universitaire de Nice 2008-07-31
NCT01254019 A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01254019 Completed GlaxoSmithKline 2013-06-28
NCT00844597 Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients https://ClinicalTrials.gov/show/NCT00844597 Completed Sarepta Therapeutics, Inc. 2010-06-30
NCT02814019 A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids https://ClinicalTrials.gov/show/NCT02814019 Active, not recruiting Santhera Pharmaceuticals 2021-08-31
NCT02579239 The Safety and Biological Activity of ATYR1940 in Patients With Limb Girdle or Facioscapulohumeral Muscular Dystrophies https://ClinicalTrials.gov/show/NCT02579239 Completed aTyr Pharma, Inc. 2016-10-05
NCT03532542 An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03532542 Enrolling by invitation Sarepta Therapeutics, Inc. 2026-08-10
NCT00773227 Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts https://ClinicalTrials.gov/show/NCT00773227 Completed Assistance Publique - Hôpitaux de Paris 2015-07-31
NCT00758225 Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) https://ClinicalTrials.gov/show/NCT00758225 Completed Santhera Pharmaceuticals 2011-01-31
NCT00654784 Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00654784 Completed Santhera Pharmaceuticals 2007-08-31
NCT00592553 Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) https://ClinicalTrials.gov/show/NCT00592553 Completed PTC Therapeutics 2009-12-31
NCT00494195 Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D) https://ClinicalTrials.gov/show/NCT00494195 Completed Nationwide Children’s Hospital 2011-06-30
NCT00451074 Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons https://ClinicalTrials.gov/show/NCT00451074 Completed Nationwide Children’s Hospital 2009-07-31
NCT00428935 Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00428935 Completed Nationwide Children’s Hospital 2009-03-31
NCT00296621 Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00296621 Completed Assistance Publique - Hôpitaux de Paris 2008-02-29
NCT00264888 Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00264888 Completed PTC Therapeutics 2007-05-31
NCT00243789 Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00243789 Completed Cooperative International Neuromuscular Research Group 2007-12-31
NCT00159250 Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00159250 Completed Imperial College London 2008-12-31
NCT00110669 High-dose Prednisone in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00110669 Completed Cooperative International Neuromuscular Research Group 2007-12-31
NCT00104078 Study Evaluating MYO-029 in Adult Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00104078 Completed Wyeth is now a wholly owned subsidiary of Pfizer NA
NCT00102453 Pentoxifylline in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00102453 Completed Cooperative International Neuromuscular Research Group 2006-07-31
NCT00005574 Gentamicin Treatment of Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00005574 Completed National Institutes of Health Clinical Center (CC) NA
NCT00033813 KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00033813 Completed Cooperative International Neuromuscular Research Group 2006-12-31
NCT00033189 An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00033189 Completed Cooperative International Neuromuscular Research Group NA
NCT00018109 A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT00018109 Completed National Center for Research Resources (NCRR) NA
NCT00027391 Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD) https://ClinicalTrials.gov/show/NCT00027391 Completed FDA Office of Orphan Products Development NA
NCT00004685 Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00004685 Completed FDA Office of Orphan Products Development NA
NCT00004646 Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00004646 Completed Office of Rare Diseases (ORD) NA
NCT00016653 Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00016653 Completed Cooperative International Neuromuscular Research Group NA

Observational Studiess

#If less then 500 studies

nct_id brief_title link overall_status source primary_completion_date
NCT03319030 Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD) https://ClinicalTrials.gov/show/NCT03319030 Completed Ann & Robert H Lurie Children’s Hospital of Chicago 2018-04-30
NCT02994030 Biomarker for Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02994030 Recruiting Centogene AG Rostock 2023-12-31
NCT02972580 Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD https://ClinicalTrials.gov/show/NCT02972580 Active, not recruiting Nationwide Children’s Hospital 2020-12-31
NCT02921321 Pilot Study of Cardiac MR in Patients With Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02921321 Recruiting Children’s National Research Institute 2023-08-31
NCT02895763 Quality of Life in Neuromuscular Disease https://ClinicalTrials.gov/show/NCT02895763 Completed CHU de Reims 2012-12-31
NCT02877784 Screening in Oculopharyngeal Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02877784 Completed University of Florida 2017-08-31
NCT02766985 Rasch-analysis of Clinical Severity in FSHD https://ClinicalTrials.gov/show/NCT02766985 Completed University of Kansas Medical Center 2017-04-18
NCT02759302 MRI on Persons With Mutations in POMT2 Gene (LGMD2N) https://ClinicalTrials.gov/show/NCT02759302 Completed Rigshospitalet, Denmark 2017-04-30
NCT02745938 GDF-15 as a Biomarker for Mitochondrial Disease https://ClinicalTrials.gov/show/NCT02745938 Completed Rigshospitalet, Denmark 2017-08-31
NCT02685215 Prognostic Factors , Morbidity and Mortality in Patients With Neuromuscular Disorders Admitted in ICU https://ClinicalTrials.gov/show/NCT02685215 Active, not recruiting Centre d’Investigation Clinique et Technologique 805 2019-12-31
NCT02635321 MRI and Muscle Involvement in Patients With Mutations in GMPPB https://ClinicalTrials.gov/show/NCT02635321 Completed Rigshospitalet, Denmark 2016-04-30
NCT02625662 Facioscapulohumeral Dystrophy in Children https://ClinicalTrials.gov/show/NCT02625662 Completed University Medical Center Nijmegen 2017-08-31
NCT02571205 Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02571205 Active, not recruiting Newcastle-upon-Tyne Hospitals NHS Trust 2019-02-28
NCT02541292 Muscle Inflammation and Fat Infiltration in Patients Affected by FSHD https://ClinicalTrials.gov/show/NCT02541292 Active, not recruiting Rigshospitalet, Denmark 2018-11-30
NCT02532244 Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases https://ClinicalTrials.gov/show/NCT02532244 Recruiting Nemours Children’s Clinic 2022-12-31
NCT02501083 Natural History of Cardiac and Respiration Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation https://ClinicalTrials.gov/show/NCT02501083 Active, not recruiting Centre d’Investigation Clinique et Technologique 805 2018-11-30
NCT02470962 Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02470962 Active, not recruiting University Children’s Hospital, Zurich 2020-04-30
NCT02466217 Phenomics in Autoimmune and Inflammatory Diseases https://ClinicalTrials.gov/show/NCT02466217 Recruiting Assistance Publique - Hôpitaux de Paris 2022-07-31
NCT02413190 Bone Health in Facioscapulohumeral Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02413190 Completed Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 2015-07-31
NCT02195999 Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02195999 Completed University of Florida 2019-03-06
NCT02194725 Where Does Hope Fit In? The Relationship Between Hope, Uncertainty, and Coping Efficacy in Mothers of Children With Duchenne/Becker Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02194725 Completed National Institutes of Health Clinical Center (CC) 2015-04-20
NCT02165358 Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I https://ClinicalTrials.gov/show/NCT02165358 Completed Rigshospitalet, Denmark 2015-01-31
NCT02159612 1 Year MRI Followup in Facioscapulohumeral Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02159612 Completed Rigshospitalet, Denmark 2015-03-31
NCT02153970 Calibration and Validation of the PROMIS and Neuro-QOL Questionnaires in Cerebral Palsy and Congenital Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02153970 Completed National Institutes of Health Clinical Center (CC) 2018-04-04
NCT02034305 Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy https://ClinicalTrials.gov/show/NCT02034305 Recruiting University of Pittsburgh 2021-01-31
NCT01990976 Study of Morphology and Functional Magnetic Resonance Imaging (MRI) Muscle Patients With Muscular Dystrophy Type FSHD Benefiting a Physical Training Introduced. https://ClinicalTrials.gov/show/NCT01990976 Completed Centre Hospitalier Universitaire de Saint Etienne 2015-06-30
NCT01950897 Muscle Tissue Bank for Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01950897 Completed Atrium Health 2015-01-30
NCT01931644 At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions https://ClinicalTrials.gov/show/NCT01931644 Recruiting Sanguine Biosciences 2025-08-31
NCT01851447 Skeletal Muscle Biomarkers in People With Fragile Sarcolemmal Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01851447 Active, not recruiting National Institutes of Health Clinical Center (CC) 2021-02-20
NCT01712152 Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study https://ClinicalTrials.gov/show/NCT01712152 Completed Hospital Rudolfstiftung 2014-04-30
NCT01689480 Prospective Study for 24-months of Physical Training Introduced in Lifestyle of Patients With FSHD : Tolerance, Sustainability and Efficiency of Unsupervised Training Program. https://ClinicalTrials.gov/show/NCT01689480 Completed Centre Hospitalier Universitaire de Saint Etienne 2015-09-30
NCT01671865 Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01671865 Active, not recruiting Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 2020-06-30
NCT01633866 Pediatric Radio Frequency Coils Generic https://ClinicalTrials.gov/show/NCT01633866 Active, not recruiting Children’s Hospital Medical Center, Cincinnati 2022-06-30
NCT01568658 Genetic and Physical Study of Childhood Nerve and Muscle Disorders https://ClinicalTrials.gov/show/NCT01568658 Recruiting National Institutes of Health Clinical Center (CC) NA
NCT01506518 Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01506518 Active, not recruiting University of North Carolina, Chapel Hill 2020-09-30
NCT01491555 Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01491555 Completed Boston Children’s Hospital 2015-09-30
NCT01459302 Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders https://ClinicalTrials.gov/show/NCT01459302 Recruiting University of Massachusetts, Worcester 2021-10-31
NCT01451281 Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01451281 Completed National Institutes of Health Clinical Center (CC) NA
NCT01437345 A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD https://ClinicalTrials.gov/show/NCT01437345 Completed Cooperative International Neuromuscular Research Group 2017-08-31
NCT01380964 Research of Biomarkers in Duchenne Muscular Dystrophy Patients https://ClinicalTrials.gov/show/NCT01380964 Completed Genethon 2015-12-31
NCT04384354 Digestive Events in Duchenne Muscular Dystrophy Patients https://ClinicalTrials.gov/show/NCT04384354 Completed Centre d’Investigation Clinique et Technologique 805 2018-12-31
NCT04369209 A Registered Cohort Study on Facioscapulohumeral Muscular Dystrophy https://ClinicalTrials.gov/show/NCT04369209 Recruiting First Affiliated Hospital of Fujian Medical University 2020-12-31
NCT04349566 Fast Troponin as a Biomarker to Assess Exercise-induced Muscle Damage in Muscle Diseases https://ClinicalTrials.gov/show/NCT04349566 Enrolling by invitation Rigshospitalet, Denmark 2020-12-31
NCT04287582 The Evaluation of Muscle Activation in Climbing up Stairs Activity in Children With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT04287582 Recruiting Hacettepe University 2020-03-07
NCT04254172 A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT04254172 Recruiting Pfizer 2021-08-12
NCT04193085 Wearable Technology to Assess Gait Function in SMA and DMD https://ClinicalTrials.gov/show/NCT04193085 Recruiting Columbia University 2020-12-31
NCT04045158 Diaphragm Ultrasound in Neuromuscular Disorders https://ClinicalTrials.gov/show/NCT04045158 Active, not recruiting Centre d’Investigation Clinique et Technologique 805 2019-12-28
NCT04035967 Investigation of Parents’ Anxiety Level and Health Related Quality of Life in Different Types of Physical Disabilities https://ClinicalTrials.gov/show/NCT04035967 Completed Sanko University 2019-05-01
NCT04012671 A Registered Cohort Study on Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT04012671 Recruiting First Affiliated Hospital of Fujian Medical University 2039-12-31
NCT03951675 The Burden of Access in Duchenne Muscular Dystrophy in the US https://ClinicalTrials.gov/show/NCT03951675 Recruiting University of Florida 2020-06-30
NCT03947112 Physical Activity Level of Norwegian Boys With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03947112 Recruiting Haukeland University Hospital 2021-03-30
NCT03930628 Limb-Girdle Muscular Dystrophy Type 2I in Norway https://ClinicalTrials.gov/show/NCT03930628 Recruiting University Hospital of North Norway 2022-09-30
NCT03882827 Natural History of Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03882827 Recruiting Genethon 2022-06-30
NCT03874910 Pathology Analysis of OPMD Patient Myotomies https://ClinicalTrials.gov/show/NCT03874910 Recruiting Hillel Yaffe Medical Center 2023-03-31
NCT03842878 Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I https://ClinicalTrials.gov/show/NCT03842878 Recruiting Genethon 2022-04-30
NCT03786913 Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases https://ClinicalTrials.gov/show/NCT03786913 Completed Benha University 2019-02-02
NCT03680365 Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families https://ClinicalTrials.gov/show/NCT03680365 Completed Jett Foundation, Inc. 2019-03-15
NCT03589573 Effect of Muscle Strength on Hamstring Flexibility in Children With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03589573 Completed Hacettepe University 2017-09-25
NCT03552874 Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03552874 Completed Hacettepe University 2017-07-31
NCT03534349 Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional Performance https://ClinicalTrials.gov/show/NCT03534349 Completed Hacettepe University 2017-06-30
NCT03521271 Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03521271 Completed Hacettepe University 2018-01-26
NCT03513367 The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM. https://ClinicalTrials.gov/show/NCT03513367 Recruiting University Hospital, Toulouse 2019-09-19
NCT03492346 Limb Girdle Muscular Dystrophy Type 2E Recruitment Study https://ClinicalTrials.gov/show/NCT03492346 Recruiting Nationwide Children’s Hospital 2020-03-28
NCT03488784 Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E https://ClinicalTrials.gov/show/NCT03488784 Recruiting Nationwide Children’s Hospital 2020-06-30
NCT03458832 Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD https://ClinicalTrials.gov/show/NCT03458832 Recruiting University of Kansas Medical Center 2022-03-31
NCT01182324 The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor https://ClinicalTrials.gov/show/NCT01182324 Completed National Institutes of Health Clinical Center (CC) NA
NCT01125709 Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS) https://ClinicalTrials.gov/show/NCT01125709 Completed Cooperative International Neuromuscular Research Group 2010-06-30
NCT01098708 This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01098708 Completed Washington University School of Medicine 2013-11-30
NCT01081080 Cardiac Magnetic Resonance in Children With Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01081080 Completed Cooperative International Neuromuscular Research Group 2011-10-31
NCT01066455 Cardiac Outcome Measures in Children With Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01066455 Completed Cooperative International Neuromuscular Research Group 2012-05-31
NCT00893334 Evaluation of Limb-Girdle Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00893334 Completed Cooperative International Neuromuscular Research Group 2013-12-31
NCT04202627 Biomarker Development in LGMD 2i https://ClinicalTrials.gov/show/NCT04202627 Enrolling by invitation Virginia Commonwealth University 2021-07-31
NCT01484678 Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy https://ClinicalTrials.gov/show/NCT01484678 Active, not recruiting University of Florida 2021-08-31
NCT03589612 Regression of Hamstring Flexibility and Performance in Children With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT03589612 Completed Hacettepe University 2019-01-24
NCT00622453 Arrhythmias in Myotonic Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00622453 Completed Indiana University 2006-01-31
NCT00457912 Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00457912 Completed Nationwide Children’s Hospital 2018-01-01
NCT00390104 Molecular Analysis of Patients With Neuromuscular Disease https://ClinicalTrials.gov/show/NCT00390104 Recruiting Boston Children’s Hospital 2025-12-31
NCT00313677 Clinical Trial Readiness for the Dystroglycanopathies https://ClinicalTrials.gov/show/NCT00313677 Recruiting University of Iowa 2020-07-31
NCT00312247 Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy https://ClinicalTrials.gov/show/NCT00312247 Completed Shriners Hospitals for Children 2014-12-31
NCT00272883 Molecular and Genetic Studies of Congenital Myopathies https://ClinicalTrials.gov/show/NCT00272883 Recruiting Boston Children’s Hospital 2050-01-31
NCT00138931 Genetics of Cardiovascular and Neuromuscular Disease https://ClinicalTrials.gov/show/NCT00138931 Recruiting University of Chicago 2025-01-31
NCT00082108 Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry https://ClinicalTrials.gov/show/NCT00082108 Recruiting University of Rochester 2023-06-30
NCT00001871 Study of Muscle Abnormalities in Patients With Specific Genetic Mutations https://ClinicalTrials.gov/show/NCT00001871 Completed National Institutes of Health Clinical Center (CC) NA
NCT00001164 Studies of Patients With Skin Disease, Patients With Neurological Degenerations, and Normal Volunteers https://ClinicalTrials.gov/show/NCT00001164 Completed National Institutes of Health Clinical Center (CC) NA
NCT00004769 Study of Muscle Wasting and Altered Metabolism in Patients With Myotonic Dystrophy https://ClinicalTrials.gov/show/NCT00004769 Completed University of Rochester 2000-03-31
NCT00004568 Study of Inherited Neurological Disorders https://ClinicalTrials.gov/show/NCT00004568 Recruiting National Institutes of Health Clinical Center (CC) NA

Registries

#If less then 500 registries

nct_id brief_title link overall_status source primary_completion_date
NCT02413450 Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias https://ClinicalTrials.gov/show/NCT02413450 Enrolling by invitation Johns Hopkins University 2023-08-31
NCT02369731 Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) https://ClinicalTrials.gov/show/NCT02369731 Recruiting PTC Therapeutics 2025-05-30
NCT02340923 A Device for Rapid, Painless, Bedside Muscle Evaluation of Children https://ClinicalTrials.gov/show/NCT02340923 Completed Skulpt, Inc. 2016-06-30
NCT02124616 National Registry for Egyptian Pediatric Neuromuscular Diseases https://ClinicalTrials.gov/show/NCT02124616 Recruiting Ain Shams University 2020-04-30
NCT02069756 The Duchenne Registry https://ClinicalTrials.gov/show/NCT02069756 Recruiting The Duchenne Registry 2027-10-31
NCT01963897 Duchenne Muscular Dystrophy < 18y in Norway: Genotype/Phenotype, Growth, Puberty, Bone Health and Quality of Life. https://ClinicalTrials.gov/show/NCT01963897 Completed Oslo University Hospital 2017-06-30
NCT01783509 Limb Girdle Muscular Dystrophy (LGMD) Natural History https://ClinicalTrials.gov/show/NCT01783509 Completed Atrium Health 2017-07-31
NCT04120168 Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease https://ClinicalTrials.gov/show/NCT04120168 Recruiting Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition 2020-03-31
NCT04020159 Global Registry for COL6-related Dystrophies https://ClinicalTrials.gov/show/NCT04020159 Recruiting Newcastle-upon-Tyne Hospitals NHS Trust 2020-10-31
NCT04001595 The Global FKRP Patient Registry https://ClinicalTrials.gov/show/NCT04001595 Recruiting Newcastle University 2020-12-31
NCT04001582 The United Kingdom Facioscapulohumeral Muscular Dystrophy Patient Registry https://ClinicalTrials.gov/show/NCT04001582 Recruiting Newcastle University 2020-12-31